hATTR-PN

The U.S. Food and Drug Administration (FDA) has lifted a clinical hold on a Phase 3 clinical trial testing the investigational gene-editing therapy, nexiguran ziclumeran…

More than half of adults with hereditary ATTR amyloidosis with cardiomyopathy (hATTR-CM), a condition marked by heart damage, also have multi-nerve damage, according to…

MRI scans detect brain changes in adults with hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) before symptoms appear, according to a study. While these changes…

A diagnosis of hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) between the ages of 30 and 60 can be profoundly life-altering. The gravity of the…

Nerve ultrasound — a scan that uses high-frequency sound waves to create real-time images of the body’s nerves — may be used to monitor signs…

Adults who carry the Val30Met, or V30M, mutation — the most common cause of early-onset hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN) — often develop heart…

Hereditary diseases not only affect the body, but they also ripple through families, shaping relationships, identities, and mental health. I know this reality all too…

RNA-targeted therapies — ones that zero in on molecules acting as messengers for genetic information in cells — are safe and effective at slowing the…

Polyneuropathy, or widespread nerve damage, occurs in both hereditary and wild-type transthyretin amyloidosis (ATTR), but the hereditary form, which includes familial amyloid polyneuropathy (FAP),…

Undergoing a liver transplant and/or receiving disease-modifying treatments significantly improves survival in people with hereditary transthyretin amyloidosis (hATTR), a group of diseases that includes hATTR…